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Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study

Sponsor
First Affiliated Hospital of Guangxi Medical University (Other)
Overall Status
Recruiting
CT.gov ID
NCT04009525
Collaborator
Peking University People's Hospital (Other), Ruijin Hospital (Other), The 923th Hospital of People's Liberation Army (Other), Fourth Affiliated Hospital of Guangxi Medical University (Other), Liuzhou General Hospital (Other), Hainan General Hospital (Other), The Affiliated Hospital Of Guizhou Medical University (Other), The First People's Hospital of Yunnan (Other)
800
Enrollment
1
Location
3
Arms
48
Anticipated Duration (Months)
16.7
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective multicenter study to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.

Condition or Disease Intervention/Treatment Phase
Phase 4

Detailed Description

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the established curative option for thalassemia major (TM). Previous study has predicted that more than 90% of TM patients can survive after allo-HSCT with a thalassemia-free survival (TFS) in around 80% of them.The purpose of this study is to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
800 participants
Allocation:
Non-Randomized
Intervention Model:
Sequential Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Actual Study Start Date :
Jun 1, 2019
Anticipated Primary Completion Date :
May 31, 2022
Anticipated Study Completion Date :
May 31, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: MSD-HSCT

matched sibling donors hematopoietic stem cell transplantation

Drug: Busulfan
Busulfan（4 mg/kg/day,4 days）
Other Names:
  • Bu

    • Drug: Cyclophosphamide
    Cyclophosphamide（50 mg/kg/day,4 days）
    Other Names:
  • Cy

    • Drug: Fludarabine
    Fludarabine（50 mg/m2/day,3 days）
    Other Names:
  • Flu

    • Drug: Thymoglobulin
    Thymoglobulin（2.5 mg/kg/day,4 days）
    Other Names:
  • ATG

    • Drug: cyclosporine A
    cyclosporine A
    Other Names:
  • CsA

    • Drug: Mycophenolate mofetil
    Mycophenolate mofetil（0.25g/day）
    Other Names:
  • MMF

    • Drug: Methotrexate
    Methotrexate
    Other Names:
  • MTX

    		•
    Experimental: URD-HSCT

    unrelated donor hematopoietic stem cell transplantation

    Drug: Busulfan
    Busulfan（4 mg/kg/day,4 days）
    Other Names:
  • Bu

    • Drug: Cyclophosphamide
    Cyclophosphamide（50 mg/kg/day,4 days）
    Other Names:
  • Cy

    • Drug: Fludarabine
    Fludarabine（50 mg/m2/day,3 days）
    Other Names:
  • Flu

    • Drug: Thymoglobulin
    Thymoglobulin（2.5 mg/kg/day,4 days）
    Other Names:
  • ATG

    • Drug: Mycophenolate mofetil
    Mycophenolate mofetil（0.25g/day）
    Other Names:
  • MMF

    • Drug: Tacrolimus
    Tacrolimus
    Other Names:
  • FK506

    • Drug: Methotrexate
    Methotrexate
    Other Names:
  • MTX

    • Drug: Ruxolitinib
    Ruxolitinib
    Experimental: haplo-HSCT

    haplo-identical hematopoietic stem cell transplantation

    Drug: Busulfan
    Busulfan（4 mg/kg/day,4 days）
    Other Names:
  • Bu

    • Drug: Cyclophosphamide
    Cyclophosphamide（50 mg/kg/day,4 days）
    Other Names:
  • Cy

    • Drug: Fludarabine
    Fludarabine（50 mg/m2/day,3 days）
    Other Names:
  • Flu

    • Drug: Thymoglobulin
    Thymoglobulin（2.5 mg/kg/day,4 days）
    Other Names:
  • ATG

    • Drug: Mycophenolate mofetil
    Mycophenolate mofetil（0.25g/day）
    Other Names:
  • MMF

    • Drug: Tacrolimus
    Tacrolimus
    Other Names:
  • FK506

    • Drug: Methotrexate
    Methotrexate
    Other Names:
  • MTX

    • Drug: Basiliximab
    Basiliximab
    Other Names:
  • Simulect

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Overall survival [2 years]

      2-years overall survival

    2. Thalassemia-free survival [2 years]

      2-years thalassemia-free survival

    Secondary Outcome Measures

    1. Engraftment [30 days]

      Myeloid engraftment at day +30

    2. Transplant Related Mortality [1 year]

      Transplant-related mortality by 1 year

    3. Cumulative Incidence of acute Graft Versus Host Disease [180 days]

      Acute graft versus host disease at day +180

    4. Cumulative Incidence of chronic Graft Versus Host Disease [2 years]

      Chronic graft versus host disease by 2 years

    5. Cumulative Incidence of Infectious Complications [2 years]

      Cumulative incidence of bacterial, fungal and viral infections by 2 years

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 20 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    1. Diagnosed with thalassemia major.

    2. Indication of hematopoietic stem cell transplantation.

    3. A cardiac ejection fraction of >50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function.

    Exclusion Criteria:

    1. Aspartate aminotransferase levels > 4-fold the upper limit of the normal range for our institution's lab criteria;

    2. Uncontrolled bacterial, viral or fungal infections;

    3. Any other restriction for transplantation.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 First Affiliated Hospital of Guangxi Medical University Nanning Guangxi China 510515

    Sponsors and Collaborators

    • First Affiliated Hospital of Guangxi Medical University
    • Peking University People's Hospital
    • Ruijin Hospital
    • The 923th Hospital of People's Liberation Army
    • Fourth Affiliated Hospital of Guangxi Medical University
    • Liuzhou General Hospital
    • Hainan General Hospital
    • The Affiliated Hospital Of Guizhou Medical University
    • The First People's Hospital of Yunnan

    Investigators

    • Principal Investigator: Yongrong Lai, MD, First Affiliated Hospital of Guangxi Medical University

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Yongrong Lai, Director of the Hematology Department, First Affiliated Hospital of Guangxi Medical University
    ClinicalTrials.gov Identifier:
    NCT04009525
    Other Study ID Numbers:
    • GX-HSCT-MT 2019
    First Posted:
    Jul 5, 2019
    Last Update Posted:
    Jul 5, 2019
    Last Verified:
    Jul 1, 2019
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Yongrong Lai, Director of the Hematology Department, First Affiliated Hospital of Guangxi Medical University
    thalassemia major
    hematopoietic stem cell transplantation
    Additional relevant MeSH terms:
    Thalassemia
    beta-Thalassemia
    Cyclosporine
    Mycophenolic Acid
    Cyclophosphamide
    Busulfan
    Methotrexate
    Fludarabine
    Tacrolimus
    Cyclosporins
    Thymoglobulin
    Basiliximab

    Study Results

    No Results Posted as of Jul 5, 2019